New Year, New NICE?

After a long process of thorough stakeholder consultations and reviews, the UK National Institute for Health and Care Excellence (NICE) published its much-coveted updated methods and processes manual and topic selection manual on January 31, 2022. The new health technology evaluations manual covers traditional technology appraisal and also encompasses diagnostic assessment, highly specialized technologies (HST), and medical technologies evaluation, which interestingly includes digital and screening tools. NICE states that the new manuals will “give patients earlier access to innovative new treatments.”

As previously reported, this change came after many years, during which time the multiple innovations that have emerged dictated a change in approach. Some of these areas include the need to adjust the appraisals to the necessities of rare and genetic disorders that require lengthier and more complex data collections; the incorporation of real-world evidence (RWE) in the review process; the evaluation of innovative treatments such as cell and gene therapies; more flexible approaches to data evaluation including from diverse sources such as patient input; and increasingly the assessment of the impact of a treatment on a patient’s quality of life, which by nature is a crucial yet unquantifiable metric.

Criticisms of previous NICE methodologies included that the highly specialized technologies evaluation for very rare diseases was too restrictive in scope, the procedures in place resulted in considerable delays, and the benefit to some patients was not properly and comprehensively evaluated.

Accordingly, the changes brought about in the new manual aim to

• adjust the weight given to health benefits in the most severe conditions (“severity modifier”) on the basis that there is increased need for treatments in those conditions, and therefore evidence of a positive effect should be evaluated differently and not be limited to end-of-life treatments
• increase and codify scope for systemic patient input to feed into the RWE that NICE will accept for, and apply in, its evaluation; this move to embracing RWE in a formal way is a major step toward facilitating the availability of treatments to rare or poorly understood diseases with patient and healthcare professional input
• understand the difficulties of evidence generation either in conditions affecting paediatric or orphan populations or for complex innovative treatments, as NICE has empowered its independent committees to factor this uncertainty into their evaluation outside of the traditional strict criteria; this will be to reduce additional barriers to innovation, ensuring that risk to patients is minimized
• clarify the criteria and principles underpinning the eligibility of treatments for very rare diseases into NICE’s HST Programme to make it more predictable and fairer; this is forecast to affect access to high-cost cell and gene therapies
• provide for the mantra of regulators and payers alike to “engage early” through the pathway to earlier engagement with NHS England and NHS Improvement and companies about commercial/managed access proposals, making it clearer when NICE can make a managed access recommendation

This is followed by a commitment from NICE to embrace a more agile methodology across all types of evaluations and adjust its methods and processes on a rolling basis through a modular approach, to reflect new needs and innovation, rather than performing more scarce whole-scale reviews.

While the pharmaceutical industry through the Association of the British Pharmaceutical Industry has generally welcomed the changes as a “move in the right direction,” the reception from medtech has been less enthusiastic.

The Association of British HealthTech Industries (ABHI) has relayed its concerns notably around the areas of cost saving, commercial and managed access schemes, and topic selection. The entry criterion on costs saving does not apply to medical devices in the same way, due to the fact that they may instead contribute to increments in benefits and costs. Equally, in ABHI’s view the commercial and managed access program should be adapted to medtech to ensure that products can be introduced in a way that evidence generation will be able to address any evidentiary uncertainty. Lastly, the MedTech Innovation Briefings are not appropriate for the assessment of whether a medtech product is appropriate for technology appraisal, which mistakenly leads to negative guidance from NICE for these products.

It remains to be seen how the new manual will be applied in practice. It is undoubtedly an ambitious move by NICE, which over the past two years has launched and participated in a number of initiatives to cement its role as an innovative force.

More specifically, through the Innovative Licensing and Access Pathway, NICE has been able to engage with stakeholders about accelerating access to promising treatments including those for rare genetic disorders. NICE has also undertaken to work closely with the NHS Genomic medicine service and other partners to identify the diagnostic and genomic testing technologies and enable this key area of medicinal science. Through the new Innovative Medicines Fund, NICE will now have the option to make recommendation for managed access to innovative treatments (including in the space of personalized medicine) outside of oncology.

Everyone’s eyes will be turned to NICE’s first few appraisals under the new system, but it is expected that NICE will continue updating its guidance and in the meantime produce faster and clearer guidance to much-needed treatments.